Regorafenib becomes First Drug used in Clinical Trial Platform to Investigate New Brain Cancer Therapies

Brain puzzle concept
Brain puzzle

Glioblastoma, also known as GBM, is a deadly brain cancer with only a few effective therapies. And while, over the last decade, much has been learned about the molecular characteristics of the disease, this knowledge has not yet been translated into clinical improvements for patients with the disease.

As part of the world’s first global adaptive clinical trial platform in glioblastoma, Glioblastoma Adaptive Global Innovative Learning Environment or GBM AGILE, a novel, multi-arm, platform trial designed to address challenges in how to best treat GBM, researchers will be able to speed the process of developing therapies for patients GBM. [1]

According to the developers of the trial, the seamless inferential design result in highly effective treatment arms proceed rapidly through the trial, enabling faster registration, regulatory review, and adoption for routine clinical care. At the same time, promising arms that do not meet criteria for the confirmatory stage exit the trial with a wealth of data to refine biomarker hypotheses and enable go/no go decisions outside of the trial. [1]

One of the treatment arms includes regorafenib (Stivarga?; Bayer) an oral multi-kinase inhibitor designed to targets angiogenic, stromal and oncogenic receptor tyrosine kinase.

Annouced earlier this week, the regorafenib arm of the platform trial has opened enrollment for patients with newly diagnosed and recurrent glioblastoma, the most aggressive and common form of primary brain cancer.[2]

The opening of the first clinical trial site, at Henry Ford Cancer Institute in Detroit, MI, marks the start of the international clinical trial program sponsored by the Global Coalition for Adaptive Research (GCAR).

Regorafenib will be the first drug to be evaluated in this trial.

Glioblastoma treatment options and patient outcomes have remained largely unchanged over several decades. [2] ninety-five percent of patients die within five years of diagnosis and more than half die within the first 15 months after diagnosis.[2]

“GBM is an aggressive brain tumor with few effective therapies. We are excited to open GBM AGILE and test new treatment options for our patients, who so desperately need them,” said Tom Mikkelsen, MD, of the Henry Ford Cancer Institute and medical director of Precision Medicine and Clinical Trials at Henry Ford Health System.?

As part of an agreement reached, Bayer will provide drug supply and support the clinical trial at sites enrolling patients in the Stivarga arm. By the end of 2019, GBM AGILE will open in over 40 academic medical centers and community-based institutions across the United States, with plans to expand across Europe, China, Canada, and Australia through 2020.

“We are excited that the regorafenib arm of the GBM AGILE trial is the first to enroll patients and are looking forward to seeing how regorafenib can potentially help these patients in need of treatment options,” said Scott Z. Fields, M.D., Senior Vice President and Head of Oncology Development at Bayer’s Pharmaceuticals Division. “Bayer actively supports the clinical research of regorafenib in a range of different tumor types to explore the potential of this drug to help even more patients in need.”

Clinical trial

A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma (GBM AGILE) – NCT03970447

Reference

[1] Alexander BM, Ba S, Berger MS, Berry DA, Cavenee WK, Chang SM, Cloughesy TF, et al. Adaptive Global Innovative Learning Environment for Glioblastoma: GBM AGILE. Clin Cancer Res. 2018 Feb 15;24(4):737-743. doi: 10.1158/1078-0432.CCR-17-0764. Epub 2017 Aug 16.

[2] Global Coalition for Adaptive Research. GBM AGILE: The Clinical Trial of the Future. Online https://www.gcaresearch.org/gbm-agile/. Last Accessed June 19, 2019.